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Pill That Hits A Cause Of Cystic Fibrosis Beats Expectations


4 Star


Pill That Hits A Cause Of Cystic Fibrosis Beats Expectations

Our Review Summary

Noteworthy is how the short piece still included:

  • better cost projections than some competitors offered
  • caveat that “all we know about the latest data is from press releases”
  • important disclosure following CF Foundation president’s quote
  • brief explanation of what 10 percentage points improvement really means in patients’ lives


Why This Matters

This story is important for a couple of reasons. New CF treatments are needed. Desperately. The announcement of positive results for a new mode of therapy is sure to cause great excitement among patients, providers, and investors.  The cystic fibrosis gene was identified in 1989 and with the identification came the hope that the mutation could in some way be manipulated or new treatments identified. VX-770 is a step in that direction. It remains to be seen if the drug will make it to market.


Does the story adequately discuss the costs of the intervention?


More explicitly than some other stories we saw, this blog included an estimate that the drug “would command a very high price, perhaps $250,000 a year.”

Does the story adequately quantify the benefits of the treatment/test/product/procedure?


Good enough.  We appreciate at least the attempt to explain that the 10 percentage points improvement in lung function “is a big deal for CF patients.”

Does the story adequately explain/quantify the harms of the intervention?

Not Satisfactory

The story quotes the CF Foundation president saying there were no significant side effects.  But even the press release reported some numbers for discontinuation rates and severe side effects, so we wish the blog had as well.

Does the story seem to grasp the quality of the evidence?


in just 369 words, the story did an adequate job evaluating the evidence, including the important caveat: “Now, to be perfectly clear, all we know about the latest data is from press releases.”

Does the story commit disease-mongering?


No disease mongering here. It was clear that this was a study in people with a specific genetic mutation that affects only about four percent of CF patients.

Does the story use independent sources and identify conflicts of interest?


Includes interview with CF Foundation president – not the most independent of sources since they have funded this work – but the story appropriately notes that the foundation will receive royalties from sales if the drug makes it to market.

The story also interviewed a biotech and drug analyst.

Does the story compare the new approach with existing alternatives?

Not Satisfactory

Nothing on existing alternatives.  We wish the story had included even one line as the NY Times did: “Two inhaled antibiotics and one drug that loosens mucus are approved to treat cystic fibrosis, but nothing that directly improves chloride ion transport.”

Does the story establish the availability of the treatment/test/product/procedure?


The story is clear that the drug is experimental, and that it hasn’t even been put before the FDA for review yet.

Does the story establish the true novelty of the approach?


Adequate job establishing the novelty of this approach for the target population.

Does the story appear to rely solely or largely on a news release?


It admits it relied on a news release.  But as we noted in our review of a competing NY Times story, while the study information is from a news release, the story did use several other sources. And, while we would normally chastise this type of reporting, we think that in this instance it is reasonable. Cystic fibrosis is a terrible disease that has defied attempts at a cure or even a satisfactory treatment. A drug that shows promise, albeit with the information dissemination coming from a press release, is newsworthy.

Total Score: 8 of 10 Satisfactory


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